Block enzymes tied to the androgen receptor, kill prostate tumors
Two enzymes from the protein disulphide isomerase (PDI) family enable prostate cancer cells to grow, survive, and resist treatment. This discovery, however, could be taken as an advantage to improve therapy for this type of tumor. Blocking the function of PDIA1 and PDIA5 leads to cancer cell death and a reduction in tumor size.

Block enzymes tied to the androgen receptor, kill prostate tumors
Two enzymes from the protein disulphide isomerase (PDI) family enable prostate cancer cells to grow, survive, and resist treatment. This discovery, however, could be taken as an advantage to improve therapy for this type of tumor. Blocking the function of PDIA1 and PDIA5 leads to cancer cell death and a reduction in tumor size.
Adcytherix raises $122M series A to develop novel ADCs
A year and half from its inception, Adcytherix SAS has raised a €105 million (US$122 million) series A, enabling it to take the lead antibody-drug conjugate (ADC) program into clinical development next year.
Esperion nominates ESP-2001 as preclinical candidate for PSC
Esperion Therapeutics Inc. has nominated ESP-2001 as a preclinical development candidate for the treatment of primary sclerosing cholangitis (PSC). The company will now begin IND-enabling studies with the aim of filing an IND application with the FDA to initiate first-in-human studies next year.
New dual DNMT and HDAC inhibitors for breast cancer treatment
Writing in the Journal of Medicinal Chemistry, researchers from Shenzhen University Medical School and collaborating institutions report the design, synthesis and biological evaluation of a series of quinoline-based hydroxamic acid derivatives as dual DNA methyltransferases (DNMT) and histone deacetylase (HDAC) inhibitors.
MFAP5: Potential biomarker of interstitial lung disease
To identify potential biomarkers of interstitial lung disease, researchers at West China Hospital of Sichuan University and Minda Hospital of Hubei Minzu University mined the Gene Expression Omnibus database of transcriptomes as well as compared tissues across healthy individuals and patients with connective tissue disease, who had interstitial lung disease or not.
Bioage Labs patents new NLRP3 inflammasome inhibitors
Bioage Labs Inc. has disclosed NLRP3 inflammasome inhibitors reported to be useful for the treatment of inflammatory disorders.
Readthrough of premature stop codons for epidermolysis bullosa
Researchers at the Keck School of Medicine at the University of Southern California explored whether CC-90009 can promote readthrough in the two types of epidermolysis bullosa.
AKR1B1 and AKR1B10 as prognostic markers in endometrial cancer
Endometrial cancer is the most prevalent gynecological malignancy in high-income countries, and the discovery of new biomarkers may aid in earlier detection and improve the prognosis of patients.

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Bioxcel Therapeutics Inc.’s phase III results in bipolar disorders or schizophrenia could bring Igalmi (BXCL-501) out of the clinic and into patients' homes for easier treatment.

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Conferences

Orchard’s OTL-104 is safe and efficacious in Crohn’s disease model
Gastrointestinal
NOD2 deficiency is involved in the pathogenesis of Crohn’s disease (CD) due to failure of gut innate immunity and loss of intestinal tissue homeostasis. Orchard Therapeutics Ltd. has presented preclinical data on OTL-104, human hematopoietic stem cell (HSC) gene therapy for the treatment of...
Selective GABA-A α3 modulator shows efficacy in essential tremor
Neurology/psychiatric
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric...
[68Ga]NOTA-R-54 arises as imaging probe for CXCR4+ tumors
New compound
CXC chemokine receptor 4 (CXCR4) is a receptor overexpressed in several tumor types and associated with tumor aggressiveness and risk of metastasis, resistance and recurrence. A novel PET radiopharmaceutical tracer and CXCR4 ligand, [68Ga]NOTA-R-54, was developed and tested for potential use in the...
Epigenetic approach shows durable PCSK9 silencing in primates
Bioengineering
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach,...

Cancer

GPC2-targeted CAR T-cells show activity against medulloblastoma
CAR T
Medulloblastoma (MB), the most common malignant pediatric brain tumor, represents about 20% of childhood central nervous system cancers.

Infection

Novel antibiotic based on dimethylcysteamine pleuromutilin
Antibiotic
As part of global efforts to develop next-generation antibiotics to overcome the growing problem of bacterial resistance to existing drugs, researchers at the Lanzhou Institute of Husbandry and Pharmaceutical Sciences prepared various derivatives of the dimethylcysteamine form of pleuromutilin, a...

CSPC Zhongqi Pharmaceutical Technology divulges new CEN inhibitors
Patents
CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co. Ltd. has synthesized prodrugs of deuterated baloxavir marboxil derivatives acting as cap-dependent endonuclease (CEN) (influenza virus) inhibitors reported to be useful for the treatment of influenza viral infections.
Dual-mechanism peptidomimetics to overcome bacterial drug resistance
In their efforts to develop next-generation antibiotics, researchers from Hunan University and collaborators designed amidated oligopyridinium peptidomimetics.

Neurology/psychiatric

Potential next-generation multi-target drugs against Alzheimer’s disease
Researchers at Indian Institute of Technology (BHU) Varanasi and collaborators have designed inhibitors of cholinesterase based on benzothiazole-phenylpiperizine as a potential treatment for Alzheimer’s disease.
AAV to deliver SynGAP1 gene against neurological disorders
Genetic/congenital
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the...
Tr1x’s TRX-319 cleared for clinical trial in progressive MS
Regulatory
Tr1x Inc. has obtained IND clearance from the FDA for TRX-319, paving the way for initiation of a phase I/IIa study in progressive multiple sclerosis (MS) early next year. TRX-319 is designed to combine targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, with the...
AAV-delivered PPT1 gene therapy shows promise for CLN1 disease
Gene therapy
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in the enzyme palmitoyl-protein thioesterase 1 (PPT1) due to biallelic loss-of-function mutations in...
Revir’s RTX-117 designated orphan drug for Charcot-Marie-Tooth disease
Regulatory
Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.

Endocrine/metabolic

EDIT-401 lowers LDL cholesterol by modulating LDLR
Conferences
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed...

Biomarkers

Study identifies three new genes linked to cleft lip, palate risk
Genetic/congenital
A recent study published in PLOS Genetics has identified several novel genetic variants associated with the risk of orofacial clefts, a common and complex congenital disability. Orofacial clefts are caused by a combination of genetic and environmental factors and can occur in isolation or...

New AI model can assign certainty, or uncertainty, to predictions
Science
A team of U.S. and South Korean researchers have developed an AI model called MSI-SEER that can not only predict microsatellite instability-high (MSI-H) tumors based on tissue slides, but also flag “what it does not know.” “Have you ever asked ChatGPT anything, and the response was, ‘I don’t...
Serum CST5 predicts anti-CD20 therapy resistance in MS
Conferences
Antibody-based therapy against CD20 has had successful results in the treatment of multiple sclerosis (MS), but some patients still have incomplete response to therapy and clinical relapse. The early identification of predictors of response is key to optimize treatment strategies. Proteomic...

Gastrointestinal

Purdue Research Foundation describes new ASGR-targeting compounds
Patents
Purdue Research Foundation has identified conjugates targeting asialoglycoprotein receptors (ASGR) that are comprised of a therapeutic or imaging agent. They are reported to be potentially useful for the diagnosis and/or treatment of nonalcoholic...
Agonist/inverse agonist combo improves primary sclerosing cholangitis
Conferences
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes.
ISM012-042 exerts protective effects in colitis model
Conferences
A new study aimed to investigate the therapeutic effect of ISM012-042 in a chronic T-cell transfer-induced colitis model in mice that mimicked Crohn’s disease.
Simcere reports preclinical data on bispecific antibody for IBD
Conferences
Jiangsu Simcere Pharmaceutical Co. Ltd. reported on SIM-0709, a bispecific antibody.
SARs to convert full cannabinoid receptor agonists to partial ones
Drug design, drug delivery and technologies
Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such...
Daewon Pharm patents new reversible H+/K+-ATPase inhibitors
Patents
Daewon Pharm Co. Ltd. has disclosed tricyclic derivatives acting as reversible H+/K+-ATPase inhibitors reported to be useful for the treatment of gastroesophageal reflux disease.

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